AQV-8741

AQV-8741 is a first-in-class, brain-penetrant oral small molecule developed by Aquinnah Pharmaceuticals for the treatment of tauopathy diseases, including Alzheimer's disease, progressive supranuclear palsy (PSP), and frontotemporal dementia (FTD). The drug employs a proprietary mechanism of action that targets the toxic interactions between pathological microtubule-associated protein tau (MAPT) and stress granules (biomolecular condensates). By modulating these condensates, AQV-8741 is designed to reduce and potentially reverse the formation and spread of tau aggregates. In preclinical studies, once-daily administration led to a 70% reduction in brain tau pathology. The compound has received backing from major pharmaceutical companies including Pfizer, AbbVie, and Takeda, and is currently undergoing Phase 1 clinical development.